Researchers Ask Hemophilia Patients to Evaluate Their Quality of Life on Study Questionnaire

According to a publication from Hemophilia News Today, an international team of researchers with members from France and the United Kingdom recently conducted a study of over 100 hemophilia patients to clinically evaluate the extent of their conditions and to assess the impact of the patients’ physical condition on their sense of well-being. The study was published in Patient Preference and Adherence.

About Hemophilia

Hemophilia is a group of conditions characterized by deficiencies of certain proteins found in the bloodstream called clotting factors. Clotting factors are a group of related proteins that contribute to a process known as the coagulation cascade.

When you cut yourself, groups of these blood clotting factors swarm the exposed opening. Through a series of cascading chemical reactions, the proteins slow bleeding, plug up the laceration, and set themselves over the site like a natural bandage. People with hemophilia could be missing any of the dozen blood clotting factors. There are several different subtypes of hemophilia, which are primarily differentiated by deficiencies in differing blood clotting factors.

Though serious external wounds pose hazards to people with hemophilia, everyday cuts and scrapes generally aren’t much of a concern. The biggest threat posed by hemophilia is the risk of internal bleeding that can occur almost completely unprompted in joints and deep in muscle tissue. A solid bump on the head could be enough to cause severe bleeding in the brain.

Hemophilia is almost always inherited through the X chromosome, though idiopathic cases of hemophilia have been known to occur with extreme rarity. Because men receive only one X chromosome from their mothers, they are much more likely than women to develop hemophilia.

Self-Assessing Through Questionnaires

The Patient Preference and Adherence study was commendably restrained in its scope and ambitions. While many studies endeavor to identify new biomarkers or determine the effectiveness of a new drug, this study merely sought to gain “health-related quality-of-life” data from hemophilia patients themselves.

The researchers’ methodology was appropriately understated — they gave out a series of questionnaires to 184 hemophilia patients from France and the United Kingdom. The questionnaires asked about patients’ general quality-of-life and their medical histories — such as if they had over two target joints or if they had histories of joint surgery.

Unsurprisingly, patients more seriously affected by the condition generally reported lower quality of life than those with only moderate forms of the disease. The results of the study, though they seem obvious, are nonetheless significant. “Everyone knows hemophilia is hard,” is not a scientific statement — but self-reported scores from a hemophilia patient’s questionnaire are hard data. Once you ask patients to evaluate their symptoms on paper, you have at least have a reference point with experimental basis.

The international team hopes that its efforts will adequately “…convey the burden of hemophilia and its management.”

What do you think of small studies like this which might be limited in scope but dense in potential? Share your thoughts with Patient Worthy!

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